StemCell Therapy

Lance was diagnosed with cerebral palsy a year ago. His family hopes non-FDA approved stem cell treatment for the disease can help him walk and talk.

CAMP HILL, Pa. A family in Cumberland County has turned to stem cells to treat their two-year-old son diagnosed with cerebral palsy. The only problem: stem cell treatment for the disease hasn't been approved by the FDA.

The day he was born, when he wheeled him down the hall and he was only a pound, and I started to cry and said, will he live? And he said, of course Hes only small," said Danielle Maxwell, Lance's mom.

The words, "he's only small," are what Lance's mom and father Rob have lived by since the day he was born. The preemie, born three months early, has been through several surgeries and complications along the way. But, Lance has always been a fighter.

Lance fought so hard just to survive the beginning of life, and come home with us," said Danielle. "And he is just so happy and loving and amazing.

About a year ago, Lance was diagnosed with cerebral palsy. Doctors told his family, he will never walk, talk or take care of himself.

We just dont believe that," said Danielle. "We dont.

Lance receives a lot of different therapies but, his parents did not want to just stop there.

We both overwhelmingly feel, he never gave up, he never gave up on us, he never gave up on himself," said Rob. "So, we owe it to him to give him the opportunity. Its really that simple, he deserves the opportunity."

Danielle began researching stem cell therapies, even speaking to doctors in countries overseas where treatment with stem cells is more readily accessible than in the U.S. The FDA has approved stem cell treatments for some conditions but not cerebral palsy. However, trials to determine the effectiveness of stem cell treatment for the disease are underway.

What weve seen is a small but real appearing improvement in motor function," said Doctor Charles Cox with University of Texas Health in Houston, began a trial in 2013 on the safety and effectiveness of banked cord blood or bone marrow stem cells in children with cerebral palsy, and is now just wrapping up the results from the trial.

The overall results of this study depend if youre a glass half full or half empty kind of person," said Dr. Cox. "It is not a compelling miraculous result. Its not, Oh my God, this child was treated and look at this profound benefit.'"

Because stem cell treatment for cerebral palsy is still in trial phases, it's not approved treatment by the FDA. However, the Maxwells did find a doctor in Harrisburg willing to transfer stem cells from a full-term baby's umbilical cord to Lance. But, since it isn't FDA approved, we were not allowed to be there to show Lance receiving the stem cells. The Maxwells are hopeful following this procedure Lance may someday walk and more importantly be able to communicate with them.

He wants to be involved," said Rob. "You can tell hes trying to communicate he just cant get over that hump. We believe stem cells could be that bridge to help him move a little faster.

Danielle says, it will take about six months to see if the stem cells will have any definitive benefits for Lance. But, already says she's seeing progress. She says Lance is not able to stand on his own.

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Cumberland County family turns to non-FDA approved stem cell treatment to help two-year-old son with cerebral palsy - FOX43.com

There are several steps in the transplant process. The steps are much the same, no matter what type of transplant youre going to have.

You will first be evaluated to find out if you are eligible for a transplant. A transplant is very hard on your body. For many people, transplants can mean a cure, but for some people, problems can lead to severe complications or even death. Youll want to weigh the pros and cons before you start.

Transplants can also be hard emotionally. They often require being in the hospital, being isolated, and theres a high risk of side effects. Many of the effects are short-term, but some problems can go on for years. This can mean changes in the way you live your life. For some people its just for a while, but for others, the changes may be lifelong.

Before you have a transplant, you need to discuss the transplant process and all its effects with your doctors. It also helps to talk to others who have already had transplants.

Its also very hard going through weeks and months of not knowing how your transplant will turn out. This takes a lot of time and emotional energy from the patient, caregivers, and loved ones. Its very important to have the support of those close to you. For example, youll need a responsible adult who will be with you to give you medicines, help watch for problems, and stay in touch with your transplant team after you go home. Your transplant team will help you and your caregiver learn what you need to know. The team can also help you and your loved ones work through the ups and downs as you prepare for and go through the transplant.

Many different medical tests will be done, and questions will be asked to try to find out how well you can handle the transplant process. These might include:

You will also talk about your health insurance coverage and related costs that you might have to pay.

You may have a central venous catheter (CVC) put into a large vein in your chest. This is most often done as outpatient surgery, and usually only local anesthesia is needed (the place where the catheter goes in is made numb). Nurses will use the catheter to draw blood and give you medicines.

If youre getting an autologous transplant, a special catheter can be placed that can also be used for apheresis to harvest your stem cells.

The CVC will stay in during your treatment and for some time afterward, usually until your transplanted stem cells have engrafted and your blood counts are on a steady climb to normal.

Younger people, those who are in the early stages of disease, or those who have not already had a lot of treatment, often do better with transplants. Some transplant centers set age limits. For instance, they may not allow regular allogeneic transplants for people over 50 or autologous transplants for people over 65. Some people also may not be eligible for transplant if they have other major health problems, such as serious heart, lung, liver, or kidney disease. A mini-transplant, described under Allogeneic stem cell transplant in Types of Stem Cell Transplants for Cancer Treatment may be an option for some of these people.

The hospitals transplant team will decide if you need to be in the hospital to have your transplant, if it will be done in an outpatient center, or if you will be in the hospital just for parts of it. If you have to be in the hospital, you will probably go in the day before the transplant procedure is scheduled to start. Before conditioning treatment begins (see section below), the transplant team makes sure you and your family understand the process and want to go forward with it.

If you will be having all or part of your transplant as an outpatient, youll need to be very near the transplant center during the early stages. Youll need a family member or loved one as a caregiver who can stay with you all the time. You and the caregiver will also need reliable transportation to and from the clinic. The transplant team will be watching you closely for complications, so expect to be at the clinic every day for a few weeks. You may still need to be in the hospital if your situation changes or if you start having complications.

To reduce the chance of infection during treatment, patients who are in the hospital are put in private rooms that have special air filters. The room may also have a protective barrier to separate it from other rooms and hallways. Some have an air pressure system that makes sure no unclean outside air gets into the room. If youre going to be treated as an outpatient, you will get instructions on avoiding infection.

The transplant experience can be overwhelming. Your transplant team will be there to help you physically and emotionally prepare for the process and discuss your needs. Every effort will be made to answer questions so you and your family fully understand what will be happening to you as you go through transplant.

Its important for you and your family to know what to expect, because once conditioning treatment begins (see the next section), theres no going back there can be serious problems if treatment is stopped at any time during transplant.

Conditioning, also known as bone marrow preparation or myeloablation, is treatment with high-dose chemo and/or radiation therapy. Its the first step in the transplant process and typically takes a week or two. Its done for one or more of these reasons:

The conditioning treatment is different for every transplant. Your treatment will be planned based on the type of cancer you have, the type of transplant, and any chemo or radiation therapy youve had in the past.

If chemo is part of your treatment plan, it will be given in your central venous catheter and/or as pills. If radiation therapy is planned, its given to the entire body (called total body irradiation or TBI). TBI may be given in a single treatment session or in divided doses over a few days.

This phase of the transplant can be very uncomfortable because very high treatment doses are used. Chemo and radiation side effects can make you sick, and it may take you months to fully recover. A very common problem is mouth sores that will need to be treated with strong pain medicines. You may also have nausea, vomiting, be unable to eat, lose your hair, and have lung or breathing problems.

Conditioning can also cause premature menopause in women and often makes both men and women sterile (unable to have children). (See Stem cell transplant and having children in Stem Cell Transplant Side Effects.)

After the conditioning treatment, youll be given a couple of days to rest before getting the stem cells. They will be given through your central venous catheter, much like a blood transfusion. If the stem cells were frozen, you might get some drugs before the stem cells are given. These drugs are used to help reduce your risk of reacting to the preservatives that are used when freezing the cells.

If the stem cells were frozen, they are thawed in warm water then given right away. There may be more than 1 bag of stem cells. For allogeneic or syngeneic transplants, the donor cells may be harvested (removed) in an operating room, and then processed in the lab right away. Once they are ready, the cells are brought in and given to you theyre not frozen. The length of time it takes to get all the stem cells depends on how much fluid the stem cells are in.

You will be awake for this process, and it doesnt hurt. This is a big step and often has great meaning for recipients and their families. Many people consider this their rebirth or chance at a second life. They may celebrate this day as they would their actual birthday.

Side effects from the infusion are rare and usually mild. The preserving agent used when freezing the stem cells (called dimethylsulfoxide or DMSO) causes many of the side effects. For instance, you might have a strong taste of garlic or creamed corn in your mouth. Sucking on candy or sipping flavored drinks during and after the infusion can help with the taste. Your body will also smell like this. The smell may bother those around you, but you might not even notice it. The smell, along with the taste, may last for a few days, but slowly fades away. Often having cut up oranges in the room will offset the odor. Patients who have transplants from cells that were not frozen do not have this problem because the cells are not mixed with the preserving agent.

Other side effects you might have during and right after the stem cell infusion include:

Again, side effects are rare and usually mild. If they do happen, they are treated as needed. The stem cell infusion must always be completed.

The recovery stage begins after the stem cell infusion. During this time, you and your family wait for the cells to engraft, or take, after which they start to multiply and make new blood cells. The time it takes to start seeing a steady return to normal blood counts varies depending on the patient and the transplant type, but its usually about 2 to 6 weeks. Youll be in the hospital or visit the transplant center daily for at least a few weeks.

During the first couple of weeks youll have low numbers of red and white blood cells and platelets. Right after transplant, when your counts are the lowest, you may be given antibiotics to help keep you from getting infections. (This is called prophylactic antibiotics.) You may get a combination of anti-bacterial, anti-fungal, and anti-viral drugs. These are usually given until your white blood cell count reaches a certain level. Still, you can have problems, such as infection from too few white blood cells (neutropenia), or bleeding from too few platelets (thrombocytopenia). Many patients have high fevers and need IV antibiotics to treat serious infections. Transfusions of red blood cells and platelets are often needed until the bone marrow starts working and new blood cells are being made by the infused stem cells.

Except for graft-versus-host disease, which only happens with allogeneic transplants, the side effects from autologous, allogeneic, and syngeneic stem cell transplants are much the same. Problems may include stomach, heart, lung, liver, or kidney problems. (Stem Cell Transplant Side Effects goes into the details.) You might also go through feelings of distress, anxiety, depression, joy, or anger. Adjusting emotionally after the stem cells can be hard because of the length of time you feel ill and isolated from others.

You might feel as if you are on an emotional roller coaster during this time. Support and encouragement from family, friends, and the transplant team are very important to get you through the challenges after transplant.

The discharge process actually begins weeks before your transplant. It starts with the transplant team teaching you and your primary (main) caregiver about:

For the most part, transplant centers dont send patients home until they meet the following criteria (Why Are Stem Cell Transplants Used as Cancer Treatment? has more information about neutrophils, platelets, and hematocrit):

If you do not meet all of these requirements, but still dont need the intensive care of the transplant unit, you might be moved to another oncology unit. When you do go home, you might need to stay near the transplant center for some time, depending on your condition.

The process of stem cell transplant doesnt end when you go home. Youll feel tired, and some people have physical or mental health problems in the rehabilitation period. You might still be taking a lot of medicines. These ongoing needs must now be managed at home, so caregiver and friend/family support is very important.

Transplant patients are still followed closely during rehab. You might need daily or weekly exams along with things like blood tests, and maybe other tests, too. During early rehab, you also might need blood and platelet transfusions, antibiotics, or other treatments. At first youll need to see your transplant team often, maybe even every day, but youll progress to less frequent visits if things are going well. It can take 6 to 12 months, or even longer, for blood counts to get close to normal and your immune system to work well. During this time, your team will still be closely watching you.

Some problems might show up as much as a year or more after the stem cells were infused. They can include:

Other problems can also come up, such as:

Your transplant team is still there to help you. Its important that you talk to them about any problems you are having they can help you get the support you need to manage the changes that you are going through. They can also help you know if problems are serious, or a normal part of recovery. The National Bone Marrow Transplant Link helps patients, caregivers, and families by providing information and support services before, during, and after transplant. They can be reached at 1-800-LINK-BMT (1-800-546-5268) or online at http://www.nbmtlink.org.

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Whats It Like to Get a Stem Cell Transplant? | American ...

There is new hope for patients with a rare autoimmune disorder. In mild cases, scleroderma causes areas of hardened skin. But in severe cases, it can also cause deadly hardening of internal organs like the lungs.

A transplant typically used to treat cancer is having remarkable results for patients who had little hope of surviving.

A year ago, Chuck Beschta couldn't walk more than a few minutes without stopping to rest.

"Just going out and doing normal activities outside raking the lawn, mowing the grass, shoveling the driveway, whatever, snow blowing those became impossible," he said.

After months of testing, he was diagnosed with severe scleroderma, which was hardening his skin. But even worse, it was hardening his lungs, making it hard to breathe.

"He was getting worse despite the best therapy we had to offer," University of Wisconsin rheumatologist Dr. Kevin McKown said.

McKown recommended a stem cell transplant newly approved for scleroderma to reboot Beschta's immune system.

"There's a process by which they try to remove the autoreactive immune cells, the cells that are caught in the immune process, and then they infuse that back in and hope that the body will basically take up and graft that immune system," McKown said.

Beschta saw almost immediate results. His skin was softer and his breathing improved. He hopes his scleroderma has been cured.

"I think we can be optimistic, and so far the people who have been followed out as far as 10 years out don't seem to be getting it back," McKown said.

Without a transplant, less than half the patients who have diffuse scleroderma and severe lung disease live 10 years past diagnosis.

Stem cell transplants are commonly used to treat leukemia and lymphoma, cancers that affect the blood and lymphatic system.

MEDICAL BREAKTHROUGHSRESEARCH SUMMARYTOPIC: NEW THERAPY FOR SCLERODERMAREPORT: MB #4698

BACKGROUND: Scleroderma is an autoimmune rheumatic disease where an overproduction of collagen produced in the body tissues causes the skin and internal organs to harden. The symptoms and effects range by person, but some common symptoms include hardened patches of skin (locations on the body vary,) painful and numb-feeling fingers and toes, and sharp internal pain in the esophagus, intestines, heart, lungs, or kidneys. Women are four times as likely to have scleroderma and the onset is between 30 and 50 years of age. However, anyone from infants to the elderly can have scleroderma. Possible risk factors include having certain gene variations as other family members, ethnic groups, exposure to certain medications or drugs, and already having another autoimmune disease, like rheumatoid arthritis, lupus or Sjogren's syndrome. (Source: https://www.scleroderma.org/site/SPageNavigator/patients_whatis.html;jsessionid=00000000.app30132b?NONCE_TOKEN=9B76519DF6B5819859319F0B63B805C9#.XheCGVVKhaQ , https://www.mayoclinic.org/diseases-conditions/scleroderma/symptoms-causes/syc-20351952 )

DIAGNOSING: A physical exam will be conducted as well as a blood test to check for elevated levels of antibodies the immune system produced. The doctor will also take a sample of skin to be tested in the lab. If there are complaints about internal pain, the doctor may run other tests, including imaging, organ function, and other blood tests. (Source: https://www.mayoclinic.org/diseases-conditions/scleroderma/diagnosis-treatment/drc-20351957 )

NEW TECHNOLOGY: A new stem cell transplant that's commonly known to treat cancer is improving the quality and quantity of life for those with scleroderma. Rheumatologists at University of Wisconsin Health tested the treatment since they have already been conducting bone marrow transplants for decades. Surgeons take out a sample of the patient's bone marrow, isolate the stem cells, and use radiation and chemotherapy to clean out their immune system. The same stem cells are later injected back into the patient's immune system with the hope that new cells will grow and the system is rid of the bad ones. The process is dangerous when the cells are taken out because the patient's immune system is more vulnerable, making infections more likely to occur. However, after four and a half years, 79% of patients that underwent the treatment were alive without serious complications compared to 50% that were treated with the original drugs. (Source: https://madison.com/wsj/news/local/health-med-fit/man-with-severe-autoimmune-disease-gets-stem-cell-transplant-at/article_7e8e17a5-21da-52f8-b728-fe584dab2b77.html)

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New technique developed to treat hardening of internal organs - WNDU-TV

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According to the latest report published by Credence Research, Inc.Anemia Treatment Drugs Market Growth, Future Prospects, and Competitive Analysis, 2018-2026,the global Anemia Treatment Drugs market was valued at USD 23,155.8 million in 2017 and is expected to grow at CAGR by 16.4 percent in the 2018 to 2026 forecast period.

Market Insights

According to the American Society of Hematology, anemia is the most common hematological disorder, affecting more than 3 million Americans every year. The global scenario is more serious because the World Health Organization states that around 1.62 billion people worldwide are affected by anemia, which is equal to 22.5 percent of the global population. In addition to the highest prevalence of anemia in pre-school children, men have the lowest prevalence. In addition, the highest number of individuals affected by pre-school anemia are non-pregnant women, which is approximately 31.2% of the total anemic population. Treatment depends specifically on the type of anemia and other complications associated with it. Currently, anemia is specifically targeted at supplements and chronic conditions are directly treated with blood transfusion, stem cell transplantation or bone marrow transplantation.

Browse Full Report Originally Published by Credence Research at https://www.credenceresearch.com/report/anemia-treatment-drugs-market

The major types of anemia that are treated with drugs include irondeficiency anemia, thalassemia, aplastic anemia, hemolytic anemia, sickle cellanemia, and pernicious anemia. In 2017, iron deficiency anemia and sickle cellanemia together dominated the market, accounting for almost 60% of the marketshare. The key factors responsible for the growth of these two types of anemiaare the highest prevalence of sickle cell anemia and aplastic anemia, and theavailable drug treatment costs more than other types. These two types of anemiaare expected to remain dominant throughout the forecast period due to theexpected market entry of more than 10 molecules during the forecast period from2018 to 2026.

The anemia drug market includes drugs such as vitamins & iron supplements, antibiotics, immunosuppressants, bone marrow stimulants, corticosteroids, gene therapy & iron chelating agents. In 2017, immunosuppressive and corticosteroids accounted for a combined market share of 58 percent due to key market driving factors such as increasing anemia prevalence, increasing anemia-related awareness at the initiative of government and non-government organizations, and continuing advances in the research and development of the anemia treatment industry. The anemia drug line is very strong and several prominent players are present along with their promising molecules. The most efficient molecules in the drug pipeline are FG-4592/roxadustat (FibroGen), Daprodustat / GSK1278863 (GlaxoSmithKline), Molidustat / BAY85-3934 (Bayer), Rivipansel (Pfizer), Luspatercept (Celgene), OMS721 (Omeros Corporation) and LentiGlobin BB305 (bluebird bio). As a result, a strong drug pipeline is expected to drive the overall market for anemia treatment drugs significantly throughout the forecast period.

Market CompetitionAssessment:

The anemia treatment drug market is expected to grow significantly in the near future and there are several companies operating in this market and expected to enter the market. The overall competitive scenario is expected to observe a paradigm shift towards gene therapy & monoclonal antibody therapies.

Access Free Sample Copy of Research Report: https://www.credenceresearch.com/sample-request/59597

Key Market Movements:

List of CompaniesCovered:

Market Segmentation:

By Anemia Type Segment

Iron deficiency anemia

Thalassemia

Aplastic anemia

Hemolytic anemia

Sickle cell anemia

Pernicious anemia

By Drug Type Segment

Supplements

Antibiotics

Immunosuppressant

Bone Marrow Stimulants

Corticosteroids

Gene Therapy

Iron Chelating Agents

By Geography SegmentType

Access Free Sample Copy of Research Report: https://www.credenceresearch.com/sample-request/59597

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Anemia Treatment Drugs Market (16.4% CAGR) 2018 to 2026: Global Industry Size, Share, Growth, Trends, and Forecast - Mobile Computing Today

CAMP HILL, PA (WPMT) A family in Cumberland County has turned to stem cells to treat their two-year-old son diagnosed with cerebral palsy. The only problem: stem cell treatment for the disease hasnt been approved by the FDA.

The day he was born, when he wheeled him down the hall and he was only a pound, and I started to cry and said, will he live? And he said, of course Hes only small, said Danielle Maxwell, Lances mom.

The words, hes only small, are what Lances mom and father Rob have lived by since the day he was born. The preemie, born three months early, has been through several surgeries and complications along the way. But, Lance has always been a fighter.

Lance fought so hard just to survive the beginning of life, and come home with us, said Danielle. And he is just so happy and loving and amazing.

About a year ago, Lance was diagnosed with cerebral palsy. Doctors told his family, he will never walk, talk or take care of himself.

We just dont believe that, said Danielle. We dont.

Lance receives a lot of different therapies but, his parents did not want to just stop there.

We both overwhelmingly feel, he never gave up, he never gave up on us, he never gave up on himself, said Rob. So, we owe it to him to give him the opportunity. Its really that simple, he deserves the opportunity.

Danielle began researching stem cell therapies, even speaking to doctors in countries overseas where treatment with stem cells is more readily accessible than in the U.S. The FDA has approved stem cell treatments for some conditions but not cerebral palsy. However, trials to determine the effectiveness of stem cell treatment for the disease are underway.

What weve seen is a small but real appearing improvement in motor function, said Doctor Charles Cox with University of Texas Health in Houston, began a trial in 2013 on the safety and effectiveness of banked cord blood or bone marrow stem cells in children with cerebral palsy, and is now just wrapping up the results from the trial.

The overall results of this study depend if youre a glass half full or half empty kind of person, said Dr. Cox. It is not a compelling miraculous result. Its not, Oh my God, this child was treated and look at this profound benefit.'

Because stem cell treatment for cerebral palsy is still in trial phases, its not approved treatment by the FDA. However, the Maxwells did find a doctor in Harrisburg willing to transfer stem cells from a full-term babys umbilical cord to Lance. But, since it isnt FDA approved, we were not allowed to be there to show Lance receiving the stem cells. The Maxwells are hopeful following this procedure Lance may someday walk and more importantly be able to communicate with them.

He wants to be involved, said Rob. You can tell hes trying to communicate he just cant get over that hump. We believe stem cells could be that bridge to help him move a little faster.

Danielle says it will take about six months to see if the stem cells will have any definitive benefits for Lance. But, already says shes seeing progress. She says Lance is not able to stand on his own

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Cumberland County family turns to non-FDA approved stem cell treatment to help two-year-old son with cerebral palsy - fox2now.com

Treatment for sickle cell disease has come a long way since the 1970s when the life expectancy of people living with it was less than 20 years.

People with sickle cell disease are not only living longer life expectancy is now 42 to 47 years of age but are enjoying a better quality of life, too.

"In the Philadelphia area, there has been great pediatric care for sickle cells disease and because of that people who have it are living very well," said Dr. Farzana Sayani, a hematologist at Penn Medicine.

Sayani is the director of a comprehensive sickle cell program focusing on adults living with the disease. Penn also has an active transition program for youth transitioning from a pediatric institution to adult care.

Sickle cell disease is an inherited red blood cell disorder that affects about 100,000 Americans.It is most often found in people of African or Hispanic descent.About 1 in 365 African-American babies are born with sickle cell disease, according to Sayani.

People who have the disease inherit an abnormal type of hemoglobin in their red blood cells, called Hemoglobin S, from both their mother and father.When only one parent has the hemoglobin S gene, a child will have the sickle cell trait, but usually does not develop the disease. But they may pass it on to their children.

Hemoglobin is the protein in the blood responsible for carrying oxygen to the rest of the body. Hemoglobin S causes red blood cells to become stiff and sickle-shaped. Instead of being round in shape, they look like crescent moons.

Sickle cells are sticky and can bind together, blocking the flow of blood and preventing oxygen from getting where it needs to go in the body. This causes sudden attacks of pain referred to as a pain crisis.

There are severaldifferent types of sickle cell disease.Hemoglobin SS, also known as sickle cell anemia, is the most common and most severe type of sickle cell disease.

Anemia occurs when red blood cells die at a rate faster than the body can replace them. Normal red blood cells generally live for 90 to 120 days. Sickled cells only live for 10 to 20 days. This shorter life-to-death cycle is harder for the body to sustain.

Another form,Hemoglobin SC, is not as severe as sickle cell anemia, but it can still cause significant complications, Sayani said.Other forms include Hemoglobin S0 thalassemia, Hemoglobin S+ thalassemia, Hemoglobin SD and Hemoglobin SE.

Sickle cell disease screening is a mandatory part of newborn screenings in Pennsylvania.

If the screening is positive, the family is informed and plugged into the health care system in order to receive the proper care.

If the disease is not diagnosed at birth, a blood test can confirm it at any age in which symptoms start to surface.

The severity of sickle cell disease can vary.

Each individual is affected differently, making it difficult to predict who will get what complications, Sayani said. That is why a comprehensive sickle cell program is so important.

Early signs include a yellowish tint to the skin or jaundice, fatigue and a painful swelling of the hands and feet.

"Young children with sickle cell disease may be tired, not eat very well and have delayed growth," Sayani said. "They may also develop anemia, be at greater risk of infection and start to experience pain crises."

Acute pain crises, also known as vaso-occlusive crises, can lead to long stays in the hospital to manage the crippling pain. Children with sickle cell disease also tend to experience delayed growth and puberty.

As a person with sickle cell disease grows older, the sickled red blood cells start to affect various organs, bones and joints.

This can lead to acute chest syndrome, which occurs when damaged lung tissues makes it difficult to breathe. Brain complications, including stroke, are possible.People with sickle cell disease are also prone to heart damage, eye problems, and infections like chlamydia, salmonella and staphylococcus. Chronic and acute pain is common.

There are different types of medicine that can help manage sickle cell disease.

Last year, an oral medicine was approved that makes sickle cells less likely to sickle. So was an intravenous medicine that has been shown to reduce pain crises and hospitalizations by 50%. Some people living with sickle cell disease also may need regular blood transfusions.

Hydroxyurea has also been used successfully for many years to reduce pain crises and the need for blood transfusions and hospitalizations.

Currently, blood and bone marrow transplant is the only way to cure the disease. But it is not an option for everyone because of the difficulty of finding a well-matched stem cell donor.

A related donor is best but only about a third of sickle cell patients have a donor that is related and fully-matched, Sayani said.

While these transplants have a 85% or more success rate, they also are associated with significant risks, including organ dysfunction, infection and graft vs. host disease which can be quite debilitating.

Transplants completed in children have the best results, Sayani said. But because of the risks involved, doctors only suggest it for patients with severe forms of the disease.

Early clinical trials with gene therapy are also showing promise, she added.

See more here:
New sickle cell disease treatments are helping people live longer and giving them a higher quality of life - PhillyVoice.com

The newly released Dish Life: The Game is described as "part Sims, part Tamagotchi". It sees you starting out as an undergraduate student, gradually rising through the scientific ranks to become a professor. You'll do this while helping your colleagues and managing the day-to-day running of your lab. It ultimately aims to showcase the laboratory as a social world, where strong relationships are key to achieving great science.

I first played developer Pocket Sized Hands' latest back in January at Pocket Gamer Connects London, and I felt it was one of the most interesting titles on the show floor. What first appeared to be a fairly straightforward educational game funded by the University of Cambridge, no less quickly revealed itself to be a surprising and layered, though still very accessible, sim with some intriguing role-playing elements.

The basic idea is that you'll work to run and expand your lab, dealing with the various complications and challenges that arise over time. Your stem cells need to be cared for, your colleagues may require your support, and you're going to have to consider a number of tough decisions that could make or break your research and friendships. You may also have to deal with things like bullying in the workplace, media controversies, and challenging ethical quandaries.

It's the sim aspects and enjoyable minutia of running a lab that makes Dish Life feel so very distinctive. Caring for your stem cells scratches that Tamagotchi itch, and managing the lab and its staff is satisfying work.

If you hear the term "educational game" and instantly switch off, know that it easily sidesteps the tedium of the genre to deliver an experience with personality to spare. There are definitely moments where your knowledge will be tested, such as the little reports you have to write, but they feel natural and dare I say it? quite fun.

It was developed in collaboration with Cambridge sociologists and stem cell scientists from the university's Stem Cell Institute, including producerDr. Lucy van de Wiel (Cambridge Department of Sociology's ReproSoc group), giving it a certain level of authenticity. The game follows on from a short film directed by Chloe Thomas and produced by Dr. Karen Jent (ReproSoc) and stem cell scientist Dr. Loriana Vitillo. I've embedded it above for you to give it a watch.

Dish Life: The Game is now available for download from both the App Store and Google Play as a free-to-play title.

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Dish Life: The Game, available now for iOS and Android, challenges you to run your very own stem cell lab - Pocket Gamer

AN EXTRAORDINARY teenager recovering from a fresh round of lifesaving treatment as she battles bone cancer is in line for the Young Scot of the year award.

Brave Molly Cuddihy from Gourock has won the hearts of people in Inverclyde and beyond as she fights the extremely rare Metastatic Ewing's Sarcoma.

The remarkable Clydeview Academy pupil has kept a smile on her face throughout her ordeal and raised an incredible 250,000 at a fundraising ball to help younger children battling cancer.

Earlier this month she underwent a stem cell transplant which left her in intensive care.

Her dad John, of Doune Gardens, said: "Molly is unable to physically speak at the moment because of side effects.

"She was so surprised by her nomination and over the moon.

"She found out on the day she went in for her transplant.

"It lifted her spirits and it lifts us all.

"Molly is incredible and an inspiration.

"She never feels sorry for herself, she just gets back up again and gets on with it.

"Again we have to thank everyone for their support - we are overwhelmed by the support of everyone out there.

"If love and kindness was a pill, Molly would be cured."

After the stem cell transplant Molly was in intensive care because of a series of complications caused by the treatment.

But her dad told the Telegraph: "The signs from the stem cell transplant are good."

Molly now faces a spell in isolation because the treatment has left her body with the protection of an unborn child.

Since her fundraising ball in October Molly has been undergoing treatment for cancer while also working with health chiefs to put plans in place for a special room in the Royal Hospital for Sick Children for children aged eight to 12 year olds.

Schoolgirl Molly was only 15 when she diagnosed with cancer in January 2018 and spent nearly a year in hospital.

She had bone cancer in her ribcage, which presented itself as a mass on her side but it had spread to her lungs and her spine.

She faced many months of gruelling chemotherapy and radiotherapy to blast the cancer.

The one in her spine is inoperable and has to be treated with radiotherapy.

On top of that there was the added complication of a bacterial infection contracted in hospital which threatened her life.

Despite all of this, during her first year in hospital Molly sat her Nationals exams in hospital and scored straight As.

In August last year she achieved straight As in her Highers and won a place at Oxford University's international summer school through all her hard work.

Weeks later Molly was told she would have to have another operation to remove one tumour, with chemo to target two more and stem cell bone marrow transplants.

During her cancer battle Molly has won the hearts of a number of celebrities, including Simon Cowell, Gary Barlow and Paulo Nutini, thanks to her fundraising exploits.

Together with her best friend Sara Millar she organised a fundraising ball last October to say thanks to all the hospital staff who have helped her.

Molly was also the first ever winner of the Rosie Mitchell Memorial Award at the Greenock Telegraph's Community Champions night last year.

Molly has now been shortlisted for Young Scotswoman of the Year hosted by Newsquest, which owns the Greenock Telegraph.

The public vote is open now and locals can back Molly up until until 5pm today.

The winner will be announced at the glittering gala in association with the St Enoch Centre, at the Grand Central Hotel on March 26.

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Brave Gourock teen battling bone cancer is in line for the Young Scot of the year award - Greenock Telegraph

Michael Schumacher turned 51 in January and as he became a year older, his fans are hoping that he is doing fine. Apparently, fans did not stop supporting him even if he not visible since his sustained massive brain injury due to a freak accident while he was skiing in December 2013.

A few weeks after the accident, Schumachers family refused to release information about the F1 champs health recovery. The family completely shut down the media so fans were deprived of information as to what is going on and any development in Schumis health. The secrecy went on for years and up to now, they remained secretive when it comes to the motorsport legends condition.

Secret treatment

It has been over six years since Micheal Schumacher had an accident and since then, some new treatments were developed. It was reported that the former sportsman tried out one of the latest procedures available today and it is called the stem cell treatment.

The Telegraph reported that Schumi was brought to Paris and admitted at the Georges-Pompidou hospital in September. The procedure was supposedly carried out by cardiovascular surgeon Philippe Menasch and it was said that one of the medical staff attested that Schumacher is conscious after the stem-cell procedure, a treatment dubbed as the pioneer in cell surgery."

It was said that Michael Schumacher stayed at the hospital for three days and on the last day, he was transported back to his home in Switzerland via ambulance. The question is - did he improved after the treatment?

How is he today?

As per The Daily Mail, even the surgeon who performed the procedure on Michael Schumacher said that he doesnt perform miracles. It appears he is saying that even though the treatment is considered a breakthrough in the medical field today, it may not work for everyone. With the racers condition, he may have developed some kind of complications that could have made his health situation worst and this treatment may have not affected Schumi as it should.

It is believed that Michael Schumacher is still paralyzed and being nursed on his bed. His movements are still limited and If there are improvements, perhaps, he could talk a bit and move some parts of his body like his head.

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Michael Schumacher reportedly underwent breakthrough treatment; Here are details of the secret procedure - EconoTimes

Heart disease is the primary cause of death worldwide, principally because the heart has minimal ability to regenerate muscle tissue. Myocardial infarction (heart attack) caused by coronary artery disease leads to heart muscle loss and replacement with scar tissue, and the heart's pumping ability is permanently reduced. Breakthroughs in stem cell biology in the 1990s and 2000s led to the hypothesis that heart muscle cells (cardiomyocytes) could be regenerated by transplanting stem cells or their derivatives. It has been 18 years since the first clinical trials of stem cell therapy for heart repair were initiated (1), mostly using adult cells. Although cell therapy is feasible and largely safe, randomized, controlled trials in patients show little consistent benefit from any of the treatments with adult-derived cells (2). In the meantime, pluripotent stem cells have produced bona fide heart muscle regeneration in animal studies and are emerging as leading candidates for human heart regeneration.

In retrospect, the lack of efficacy in these adult cell trials might have been predicted. The most common cell type delivered has been bone marrow mononuclear cells, but other transplanted cell types include bone marrow mesenchymal stromal cells and skeletal muscle myoblasts, and a few studies have used putative progenitors isolated from the adult heart itself. Although each of these adult cell types was originally postulated to differentiate directly into cardiomyocytes, none of them actually do. Indeed, with the exception of skeletal muscle myoblasts, none of these cell types survive more than a few days in the injured heart (see the figure). Unfortunately, the studies using bone marrow and adult resident cardiac progenitor cells were based on a large body of fraudulent work (3), which has led to the retraction of >30 publications. This has left clinical investigators wondering whether their trials should continue, given the lack of scientific foundation and the low but measurable risk of bleeding, stroke, and infection.

Additionally, investigators have struggled to explain the beneficial effects of adult cell therapy in preclinical animal models. Because none of these injected cell types survive and engraft in meaningful numbers or directly generate new myocardium, the mechanism has always been somewhat mysterious. Most research has focused on paracrine-mediated activation of endogenous repair mechanisms or preventing additional death of cardiomyocytes. Multiple protein factors, exosomes (small extracellular vesicles), and microRNAs have been proposed as the paracrine effectors, and an acute immunomodulatory effect has recently been suggested to underlie the benefits of adult cell therapy (4). Regardless, if cell engraftment or survival is not required, the durability of the therapy and need for actual cells versus their paracrine effectors is unclear.

Of particular importance to clinical translation is whether cell therapy is additive to optimal medical therapy. This remains unclear because almost all preclinical studies do not use standard medical treatment for myocardial infarction. Given the uncertainties about efficacy and concerns over the veracity of much of the underlying data, whether agencies should continue funding clinical trials using adult cells to treat heart disease should be assessed. Perhaps it is time for proponents of adult cardiac cell therapy to reconsider the approach.

Pluripotent stem cells (PSCs) include embryonic stem cells (ESCs) and their reprogrammed cousins, induced pluripotent stem cells (iPSCs). In contrast to adult cells, PSCs can divide indefinitely and differentiate into virtually every cell type in the human body, including cardiomyocytes. These remarkable attributes also make ESCs and iPSCs more challenging to control. Through painstaking development, cell expansion and differentiation protocols have advanced such that batches of 1 billion to 10 billion pharmaceutical-grade cardiomyocytes, at >90% purity, can be generated.

Preclinical studies indicate that PSC-cardiomyocytes can remuscularize infarcted regions of the heart (see the figure). The new myocardium persists for at least 3 months (the longest time studied), and physiological studies indicate that it beats in synchrony with host myocardium. The new myocardium results in substantial improvement in cardiac function in multiple animal models, including nonhuman primates (5). Although the mechanism of action is still under study, there is evidence that these cells directly support the heart's pumping function, in addition to providing paracrine factors. These findings are in line with the original hope for stem cell therapyto regenerate lost tissue and restore organ function. Additional effects, such as mechanically buttressing the injured heart wall, may also contribute.

Breakthroughs in cancer immunotherapy have led to the adoption of cell therapies using patient-derived (autologous) T cells that are genetically modified to express chimeric antigen receptors (CARs) that recognize cancer cell antigens. CAR T cells are the first U.S. Food and Drug Administration (FDA)approved, gene-modified cellular pharmaceutical (6). The clinical and commercial success of autologous CAR T cell transplant to treat B cell malignancies has opened doors for other complex cell therapies, including PSC derivatives. There is now a regulatory path to the clinic, private-sector funding is attracted to this field, and clinical investigators in other areas are encouraged to embrace this technology. Indeed, the first transplants of human ESC-derived cardiac progenitors, surgically delivered as a patch onto the heart's surface, have been carried out (7). In the coming years, multiple attempts to use PSC-derived cardiomyocytes to repair the human heart are likely.

What might the first human trials look like? These studies will probably employ an allogeneic (non-self), off-the-shelf, cryopreserved cell product. Although the discovery of iPSCs raised hopes for widespread use of autologous stem cell therapies, the current technology and regulatory requirements likely make this approach too costly for something as common as heart disease, although this could change as technology and regulations evolve. Given that it would take at least 6 months to generate a therapeutic dose of iPSC-derived cardiomyocytes, such cells could only be applied to patients whose infarcts are in the chronic phase where scarring (fibrosis) and ventricular remodeling are complete. Preclinical data indicate that chronic infarcts benefit less from cardiomyocyte transplantation than do those with active wound-healing processes.

Adult cells from bone marrow or the adult heart secrete beneficial paracrine factors but do not engraft in the infarcted heart. Pluripotent stem cells give rise to cardiomyocytes that engraft long term in animal models, beat in synchrony with the heart, and secrete beneficial paracrine factors. Long-term cardiomyocyte engraftment partially regenerates injured heart, which is hypothesized to bring clinical benefits.

The need for allogeneic cells raises the question of how to prevent immune rejection, both from innate immune responses in the acute phase of transplantation or from adaptive immune responses that develop more slowly through the detection of non-self antigens presented by major histocompatibility complexes (MHCs). A current strategy is the collection of iPSCs from patients who have homozygous MHC loci, which results in exponentially more MHC matches with the general population. However, studies in macaque monkeys suggest that MHC matching will be insufficient. In a macaque model of brain injury, immunosuppression was required to prevent rejection of MHC-matched iPSC-derived neurons (8). Similarly, MHC matching reduced the immunogenicity of iPSC-derived cardiomyocytes transplanted subcutaneously or into the hearts of rhesus macaques, but immunosuppressive drugs were still required to prevent rejection (9).

Numerous immune gene editing approaches have been proposed to circumvent rejection, including preventing MHC class I and II molecule expression, overexpressing immunomodulatory cell-surface factors, such CD47 and human leukocyte antigen E (HLA-E) and HLA-G (two human MHC molecules that promote maternal-fetal immune tolerance), or engineering cells to produce immunosuppressants such as programmed cell death ligand 1 (PDL1) and cytotoxic T lymphocyteassociated antigen 4 (CTLA4) (10). These approaches singly or in combination seem to reduce adaptive immune responses in vitro and in mouse models. Overexpressing HLA-G or CD47 also blunts the innate natural killer cellmediated response that results from deleting MHC class I genes (11). However, these manipulations are not without theoretical risks. It could be difficult to clear viral infections from an immunostealthy patch of tissue, and possible tumors resulting from engraftment of PSCs might be difficult to clear immunologically.

Ventricular arrhythmias have emerged as the major toxicity of cardiomyocyte cell therapy. Initial studies in small animals showed no arrhythmic complications (probably because their heart rates are too fast), but in large animals with human-like heart rates, arrhythmias were consistently observed (5, 12). Stereotypically, these arrhythmias arise a few days after transplantation, peak within a few weeks, and subside after 4 to 6 weeks. The arrhythmias were well tolerated in macaques (5) but were lethal in a subset of pigs (12). Electrophysiological studies indicate that these arrhythmias originate in graft regions from a source that behaves like an ectopic pacemaker. Understanding the mechanism of these arrhythmias and developing solutions are major areas of research. There is particular interest in the hypothesis that the immaturity of PSC-cardiomyocytes contributes to these arrhythmias, and that their maturation in situ caused arrhythmias to subside.

A successful therapy for heart regeneration also requires understanding the host side of the equation. PSC-derived cardiomyocytes engraft despite transplantation into injured myocardium that is ischemic with poor blood flow. Although vessels eventually grow in from the host tissue, normal perfusion is not restored. Achieving a robust arterial input will be key to restoring function, which may require cotransplanting other cell populations or tissue engineering approaches (13, 14). Most PSC-mediated cardiac cell therapy studies have been performed in the subacute window, equivalent to 2 to 4 weeks after myocardial infarction in humans. At this point, there has been insufficient time for a substantial fibrotic response. Fibrosis has multiple deleterious features, including mechanically stiffening the tissue and creating zones of electrical insulation that can cause arrhythmias. Extending this therapy to other clinical situations, such as chronic heart failure, will require additional approaches that address the preexisting fibrosis. Cell therapy may again provide an answer because CAR T cells targeted to cardiac fibroblasts reduced fibrosis (15).

Developing a human cardiomyocyte therapy for heart regeneration will push the limits of cell manufacturing. Each patient will likely require a dose of 1 billion to 10 billion cells. Given the widespread nature of ischemic heart disease, 105 to 106 patients a year are likely to need treatment, which translates to 1014 to 1016 cardiomyocytes per year. Growing cells at this scale will require introduction of next generation bioreactors, development of lower-cost media, construction of large-scale cryopreservation and banking systems, and establishment of a robust supply chain compatible with clinical-grade manufacturing practices.

Beyond PSC-cardiomyocytes, other promising approaches include reactivating cardiomyocyte division and reprogramming fibroblasts to form new cardiomyocytes. However, these approaches are at an earlier stage of development, and currently, PSC-derived cardiomyocyte therapy is the only approach that results in large and lasting new muscle grafts. The hurdles to this treatment are known, and likely addressable, thus multiple clinical trials are anticipated.

Acknowledgments: C.E.M. and W.R.M. are scientific founders of and equity holders in Sana Biotechnology. C.E.M. is an employee of Sana Biotechnology. W.R.M. is a consultant for Sana Biotechnology. C.E.M. and W.R.M. hold issued and pending patents in the field of stem cell and regenerative biology.

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Stem cells and the heartthe road ahead - Science Magazine

The first time Bill Cronin Googled his own cancer diagnosis in 2016, his heart sank. He had Szary syndrome, a rare and aggressive form of cutaneous T-cell lymphoma and staring back at him were countless articles predicting a negative prognosis.

However, after receiving a stem-cell transplant at Dana-Farber/Brigham and Womens Cancer Center, Cronin is returning to the life he enjoyed before cancer.

Im at a place I never thought Id get to, Cronin says.

In 2015, Cronin, then 60, started feeling incredibly itchy and developed an accompanying rash. He went to his dermatologist, who diagnosed him with eczema and told him to return in five months. The rash continued to grow, however, and at the five month mark, Cronins dermatologist encouraged him to undergo further testing at Dana-Farber.

A blood test revealed that Cronins T-cells a type ofwhite blood cells that make up part of the immune system had becomecancerous. In the case of Szary syndrome, lymphoma cells will circulatethrough the blood stream and deposit in different areas of the skin. This willgenerally lead to a full-body rash and intense itchiness.

Cronin would need a stem cell transplant to combat the disease, but before he could receive one, his care team had to get him into remission. Patients who do not achieve remission prior to transplant have a high chance of relapsing.

When they first told me everything, I was really scared, says Cronin. But I knew I was in one of the best places in the world to figure out and treat this rare disease.

Cronins pre-transplant care was spearheaded by oncologists David Fisher, MD, and Nicole LeBoeuf, MD, MPH, clinical director of Cutaneous Oncology at Dana-Farber, with his transplant conducted by Corey Cutler, MD, MPH, medical director of the Adult Stem Cell Transplantation Program at Dana-Farber. Initially, Cronins disease was incredibly resistant; for nearly three years, mainstay drugs including steroids, monoclonal antibodies, and enzyme blockers all failed to put his disease into remission.

Ultimately, it would take a new drug, mogamulizumab (a type of immunotherapy that directly kills T-cells involved with Sezary Syndrome) to get Cronins disease into remission.

In May 2019, Cronin was cleared to undergo an allogeneic transplant, a type of transplant that uses a donors stem cells, in this case, Cronins brother. Since his transplant Cronin has remained in remission.

We had to use all of our big guns to get him totransplant, but Im pleased with where we are now, says Cutler.

I know the situation can always change, but it was great tobe able to share some good news with my family and friends, adds Cronin.

Patients like Cronin serve as a reminder of how stem cell transplants have improved and continue to impact patient outcomes, Dana-Farber experts note. Initially offered to only an incredibly small patient population when first performed at Dana-Farber in the 1970s, research advancements have, and continue to, broaden who is eligible for a transplant. In 2019, Dana-Farber/Brigham and Womens Cancer Center (DF/BWCC) surpassed 10,000 total adult transplants.

This milestone indicates our success as a program and our volume has allowed us to do the research to help move the field forward rather impressively, says Joseph Antin, MD, chief emeritus of Adult Stem Cell Transplantation at DF/BWCC.

In 1996, Dana-Farber Cancer Institute and Brigham and Womens Hospital merged their then separate transplant centers. By pooling together physical and intellectual resources, the new combined program was able to more than double the number of transplants each hospital could perform individually.

We always felt collaboration was better than competition, explains Robert Soiffer, MD, vice chair of Medical Oncology for Hematological Malignancies and chief of the Division of Hematologic Malignancies, who oversaw the merger with Antin. Each side could learn from the other, and that helped to catapult us into the leadership position we have today.

The Stem Cell Transplantation Program is also bolstered by the Connell and OReilly Families Cell Manipulation Core Facility (CMCF), which was established in 1996. The state-of-the-art center, led by Jerome Ritz, MD, not only processes the stem cells for transplant; it also assists researchers in developing new cell-based therapies for patients.

Another key component to the programs success has been the creation of the Ted and Eileen Pasquarello Tissue Bank. The Pasquarello Tissue Bank receives, processes, banks, and distributes research samplesof blood, bone marrow, and other tissues. Through a database overseen by Vincent Ho, MD, the Institute is able to log, assess, and later review every patients disease, including all complications and mutations. This technology allows researchers to explore the genetic makeup of past donors and better understand why a transplant was or was not successful.

Were still learning from biological specimens we collected 20 years ago, and it will continue to impact care 20 years from now, Soiffer says.

Today, there is a continuous push to develop new and more precise therapies to complement and improve stem cell transplants. The hope is to bring new treatment options to patients like Cronin who are facing rare and difficult diseases.

Before his diagnosis, Bill, and Barbara Finney, his partner ofnearly 30 years, were avid English Country dancers. English Country dancingevolved from the court dances of Europe in the early 17th century, and Croninand Barbara have friends from all over the country who share their passion forit.

While Cronin isnt dancing just yet, as hes stillrecovering from his transplant, he says he couldnt have gotten through thiswithout his partner on the dance floor and in life.

Barbara has been amazing and has helped take care ofeverything I couldnt do, he adds. Ive been fortunate and privileged to notonly have her, but to have been able to come to Dana-Farber.

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Transplant for Szary Syndrome is Patient's First Step in Returning to the Dance Floor - Dana-Farber Cancer Institute

Last month, a letter dropped onto Robert Jones doormat containing just about the best possible news.

It revealed the person he had donated bone marrow to was doing very well, two years on from a life-saving blood stem cell transplant.

The Pontypridd hooker doesnt know much about the recipient, other than hes a 24-year-old young man from Ireland.

But with two years having passed, that person will now receive details of his donor and can get in touch with him and meet up if he so wishes.

Its a meeting Jones would love to see take place.

He has decided to talk about what he did and the happy outcome in order to encourage more people to join the donor list.

So how did it all come about?

I have given blood for about 10, 12 years now, he explained.

Then about four years ago, the Welsh Blood Service just asked me if I would mind going on the bone marrow donor list.

I lost my grandfather, Howard, to leukaemia six years ago. So as soon as they said it could benefit people who suffer with blood cancer, I couldn't say no, really.

I joined the register and they just took an extra sample of blood to test.

I didnt expect to hear anything, but within a year and a half, Welsh Blood contacted me to say they had a potential match. I was a bit shell-shocked.

I went down and had a discussion with them. I didnt know anything about who I was going to be donating to at that point.

I went for a medical, a full body MOT to make sure I was fit enough.

With that box ticked, it was then a question of deciding which procedure to go for.

They give you two options, he explained.

Either a stem cell transplant where you are hooked up to a machine for 12 hours or taking the bone marrow, which involves drilling into your hip bone.

I went for the drilling one.

In simple terms, the stem cell is quantity over quality, whereas the bone marrow is quality over quantity.

When I had my meetings with Welsh Blood to make my choice, they said it was a 22-year-old young boy from Ireland who had leukaemia.

At the time, my brother was 23, 24 and I just thought imagine that was my brother.

The only thing I could think of was imagine the shoe was on the other foot and it was one of my family members or friends suffering.

Plus the idea of being hooked up to a machine for 12 hours straight would have skulled me to death!

So I went for the bone marrow option. It was more likely to be helpful and useful.

That was in January 2018. When you hear exactly what the procedure involved, you have nothing but admiration for Jones.

They drill straight into your hip bones to the core and then they withdraw your whole bone marrow, he said.

There were six drill holes right in the bottom of my back. It depends on the individual, the amount of marrow they take.

If theres enough to take, they keep taking it really. The nurse said they took a hell of lot more from me than a normal person, possibly because I am so big!

I think it was 600 mill they had out of me.

The operation lasted about three hours in all.

I woke up from the anaesthetic about 1pm in the afternoon and I was in so much pain I said put me straight back under!

It was such a severe ache I just couldnt ignore it. So they put more morphine in and I went straight back to sleep.

Then I woke up about 6pm and my mother was there to pick me up.

She said I just looked horrendous, as white as a ghost.

But I went home that evening, so I was in and out of hospital in the day.

I couldnt sit down for a week. I had to lie flat. So I was more or less settee or bed bound.

Obviously I was in a lot of pain and I was off work for three or four months. They were really supportive.

I was still aching for up to a year, but not to the extent as when I woke up.

Jones, who works as an area manager for Wales & West Utilities gas distribution network, obviously had a break from rugby while he recovered - although not for too long.

He was with Cross Keys at the time, having joined them following a seven-year stint with his hometown club Treorchy, for whom he played more than 200 games.

One of the big questions I asked was whether it would prevent me playing rugby, but they said it would have no long-lasting effect, he said.

I was back training after about eight weeks and played my first game after around four months, towards the end of the season.

For about a year, every time I was having scrummaging sessions or a game, the bottom of my back was just aching.

I am a hooker, so I am right in the mix of it. The second rows were pushing right on the sore spot at the bottom of my spine.

"I'm over all that now and fortunately I am still able to play, but even if it had been the end of my rugby career, that's nothing to me really.

"I've still got my good health and I'm fortunate to be still here, with all my friends and family, and that young man wouldn't be, so I have no regrets at all and if I could do it again tomorrow, I would."

A year on from his procedure, Jones received notification the recipient had survived the transplant and last month there was an even more positive update.

The letter from Welsh Blood said the young man from Ireland was very well and had not suffered any major complications.

That makes it all worthwhile, said Jones.

Its very rewarding.

And it also raises the possibility of donor and recipient meeting up.

After two years, they get all my information, so they can contact me, he said.

If he wants to get in touch with me, I would love to hear from him, just to see how he is and meet him possibly. That would be phenomenal.

I am open to the idea of getting to know him, but obviously its totally down to the individual.

Now 29, Jones is in his second season with Pontypridd having switched to Sardis Road after two years with Cross Keys.

Im loving it there. They are a really great bunch of boys, he said.

Hes currently sidelined with a hamstring problem, but understandably such issues are pretty minor in the general scheme of things when you have been through the kind of experience he has.

Ask him whether he would do it all again if he had his time over, and he provides an emphatic answer.

100 per cent. I would do it again tomorrow, he said.

I have given that person at least two years extra.

The bone marrow is the last straw. If he hadnt had that, he probably wouldnt have been around today.

"So no regrets at all. You just give someone that little hope and I would definitely encourage people to think about going on the register.

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The selfless act of popular Welsh rugby player who risked his career to save the life of a stranger - Wales Online

Care plan or support can be defined as the method followed to provide physical and clinical support to an individual with health complications. For a proper care plan, it is essential to have conceptual clarity about the physiological status of the individual. Depending on the condition of the diseases, care planning also alters.

Without access to quality healthcare, people are subjected to various types of risks. From malnutrition to pregnancy, all can become quite dangerous without proper health and support. Intake of a proper diet and exercising regularly are some of the ways through which we can stay healthy.

How To Improvise Regular Health System:

1. The importance of visual observation

Visual observation is needed to notice on the patient in the slightest uneasiness of the attitude. Visual observation is equally important in eye care too. With visual observation, an expert ophthalmologist can know the problematic areas of your eyes. To be precise, visual observations should form an integral part of a healthcare practitioners core skills. Recording what was being observed can shed light on the progress made by the patients. Visual observation also helps to demonstrate areas of potential concerns.

Assessing the routine measures that provide information about body functioning

Some common illnesses associated with the eye

Age-Related Macular Degeneration

Reasons for taking Measures

To ensure that there is no harm to the retina.

2. BMI

The measurement of a persons Body Mass Index identifies the persons weight and height. BMI gives an indicator of body fat for most of the people that can go to health-related problems. BMI means Body Mass Index that is the ratio between weight and height.

3. Heart rate

Heartbeat means the speed of the heartbeat that is measured by the number of heart diastole and systole. The heart pumps during systematic gaps. Some activities can change the rates that are strenuous jobs, exercise, and tension. The usual rate of an adult can be 60 to 100 bpm. A lower rate can mean circulatory fitness.

4. Blood Pressure

It is vital for an adult blood pressure that is (120/80). It is the ideal blood pressure. A sphygmomanometer is to estimate blood pressure. The flow of blood through the blood vessel is measured in this way. Blood pressure depends on cholesterol in the blood as well.

Assessing the functioning of care planning and individuals:

Another example is the heart rate, which indicates the blood flow and ability of the heart to facilitate the blood flow throughout the body. However, during the stage of low blood circulation with a risk of myocardial infarction, angioplasty is recommended to increase the diameter of blood vessel coming out of the heart to improve the blood flow. On the other hand, if the expansions of vessels are more than the required level, it can lead to reduced blood pressure and declined blood flow to the body. Therefore it is essential while planning for care to know the blood pressure and blood flow rate, and accordingly, they make plans.

Precisely, there is a particular checklist for preparing a care plan involving the maintenance of a clinical summary of an individual. Keeping a record for unmet support practice and its impact on the patient is imperative. These practices for care plans can profoundly help in the formulation of effective care plans.

Comparing structural and functional alterations of the eye with advancing age:

With advancing age, one would encounter a variety of eye-related issues. The common age-related eye problems include glaucoma, presbyopia, age-related macular degeneration, and retinal problems. Thus, if you want to prevent your eye problems, never forget to visit a good eye care center like Eye7 Chaudhary Eye Centre, where experts are available to take care of you.

Aging is an inevitable phenomenon for all the cells in the body leading to alterations in connective tissues, nerve tissues, connective, and epithelial tissues. Cells become larger with age, less flexible, and lesser capabilities of cell division. Therefore, the possibility of producing new cells at the elderly age is a less likely incident except for the stem cells. Most of the cells at the old age stops functioning or acts abnormally. When a human body ages, it is generally observed that the skin becomes pale and the hair turns grey. In addition to that, the body function slows down.

Starting from childhood, connective tissues such as bone and blood vessels grow till old age and then become stiff. Usually, there are three main types of connective tissues. Further, the structure of cell membranes alters and becomes less active in procuring oxygen and energy from food. Body cells also lose their capability to expel carbon dioxide as well.

As the cells and tissues change, they exert their cumulative effect on the entire organ by changing their structure and functional abilities. At the age of 20 years, the heart muscle can pump out blood at a rate of 10 times higher than is needed for keeping the body alive, and this efficiency declines by 1% each year.

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The Physiological Principles in Health and Social Care One Must Know - Times of India

Over the past 15 years, Californias stem cell program has funded over 1,000 research, training and community engagement projects focused on stem cell technology in the state.

But in October of last year, the agency in charge of administering the states stem cell program awarded its last round of new funds, allocating the last of a total of $3 billion in funding approved by California voters. Now, the author of Proposition 71, which established the program in 2004, is seeking another round of $5.5 billion in funding for stem cell research in a measure vying for a spot on the Nov. 5 ballot.

The scientists and patient advocates in California have proven through the California stem cell initiative funding that they can change the future of medicine and human suffering, the measures author, Robert Bob Klein II told the Business Journal. California funding has filled the gap of the federal governments failure to fund this revolution in medicine.

Kleins interest in stem cell research and regenerative medicine arose from his youngest sons ultimately fatal battle with diabetes. Diagnosed with Type I Diabetes at age 11, Kleins son Jordan required human insulin-producing beta cells, the artificial production of which to the best knowledge of the scientific community at this point requires stem cells.

In 2016, 26-year-old Jordan Klein died of complications related to the disease, two years after scientists first made significant progress on finding a treatment developed with the help of human embryonic stem cells.

Klein blames the federal governments resistance to embracing stem cell research for the lack of adequate treatment options that lead to his sons death. My youngest son died. If they hadnt held it up in D.C., he would be alive, he said. How many children, how many adults are going to die before they create enough stability to advance therapies that mitigate or cure these chronic diseases?

For years, Klein a wealthy real estate developer had tried to affect change on the federal level, before shifting his attention to his home state. I became focused on what California could do, because the federal government was encumbered by religious issues, he explained.

Despite having no experience in the field of scientific research, Klein was able to mount a successful campaign, with 59.1% of California voters approving the creation of a state-funded stem cell program and an agency to govern it. The California Institute of Regenerative Medicine held its first meeting in December 2004 and issued its first round of funding in 2006, after battling several challenges to the proposition in state and federal court.

Since then, CIRMs funding has enabled conferences, translational research projects and clinical trials exploring cures for various types of cancers, diabetes and neurological disorders. While a majority of funding went to the University of Californias main campuses as well as regenerative medicine companies and institutes in major hubs like San Diego and the Bay Area, California State University, Long Beach and Torrance-based Pathways to Stem Cell Science have also received funds from CIRM.

CIRM has created a new industry in California that has been tremendously beneficial for the California economy, Dr. Victoria Fox, president of Pathways, told the Business Journal. Her company, which offers stem cell extraction and education services, has received both direct and indirect funding from CIRM.

Last year, Pathways hosted the SPARK Annual Meeting, an event that highlights the scientific accomplishments of students in the SPARK high school summer training program funded through a CIRM grant. Foxs company also provides educational services through CIRMs Bridges program at CSULB, in which students from state and junior colleges compete for six-month lab positions at UC research labs.

[These programs] are important to us, not only because they generate income to operate, but because they generate a workforce, Fox explained.

She said other companies often approach her in search of stem cell scientists. If voters decided not to extend funding for CIRM and its program in November, Fox said shed be hard-pressed to find an equivalent network for talent acquisition. I dont know where Im going to refer companies to find talent. I really dont know.

Dr. Aaron Levine, an associate professor at the School of Public Policy at Georgia Tech, has focused primarily on the intersection between public policy and bioethics. In this context, Levine has followed and reviewed Californias stem cell program for years, and he agreed that its impact has been transformative.

CIRM stepped in to fill a gap when the National Institutes of Health was restricting its funding in this space, Levine said. The research that CIRM has supported, as well as the training programs, has had quite a big impact on the field.

But Levine also pointed to what he described as missed opportunities, as the program enters its bid for renewal. One example is the new proposals requirement to commit a certain percentage of funds to finding cures for specific diseases, such as Alzheimers and other neurological disorders.

From a science policy perspective, I dont really like that. I would like the funding agency to have the flexibility to fund the best science they see submitted, the most promising science, Levine said. Klein argued that setting aside a certain percentage of funds for research on neurological diseases will ensure that they wont be left behind in favor of less costly research projects.

Levine also noted that the program has yet to resolve one crucial question: Who will pay for patients treatment with costly stem cell therapies once theyre ready to hit the market? Per-patient costs for stem cell therapies can easily reach several hundred thousand dollars and as research advances, more patients are expected to qualify.

Suddenly, thats just such a substantial sum of money that it becomes a fundamental challenge to how we pay for healthcare, how we pay for medicine in the United States, Levine said. Subsidies for California residents, whose taxes helped pay for the research necessary to bring these cures and therapies to market, would be one option, Levine noted.

Despite these concerns, Levine said he supports the measure to extend the program. Even though this is not the perfect measure, I think theres a lot of value in CIRM and it makes sense to continue it, he said. In the end, it will be up to California voters to decide.

It largely will rise and fall on whether theres a motivated campaign for and against it and what people whove never really thought about stem cell research as a state ballot issue are going to think about this particular initiative when it comes in the fall, Levine said.

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Should California invest another $5.5 Billion into stem cell research? - Long Beach Business Journal - Long Beach News

NEW YORK, Feb. 14, 2020 /PRNewswire/ -- JDRF, the leading global organization funding type 1 diabetes (T1D) research, funded $121.5 million directly and helped generate more than $400 million in total T1D research funds from nonprofits, government, and industry to propel a year of remarkable breakthroughs in 2019, JDRF announced today. JDRF research funding increased by 10 percent over the previous year.

"I am more excited about our progress than ever before, given the tremendous breakthroughs made in 2019," said Aaron J. Kowalski, Ph.D., president and CEO of JDRF. "We could not be driving this work forward without the support of so many dedicated individuals, partners and organizations all helping us deliver on our mission to cure T1D and improve lives."

JDRF funding supported more than 180 active T1D research grants and is funding about 70 clinical trials for drugs, biologic and devices to prevent or cure T1D. The organization also supported new investment through the JDRF T1D Fund in 15 T1D companies, including 11 focused on cures.

"We are focused on beta cell therapies and immune therapies to deliver cures for T1D," Kowalski said. "At the same time, we continue accelerating work in glucose control and complication therapies to improve the lives of those living with type 1 diabetes today."

Highlights of breakthroughs outlined in the 2019 Annual Report are:

Progress Toward Cures:

Improving Lives:

Reducing Complications:

JDRF's Annual Report also cites progress in advocacy and community engagement, two areas aimed at both supporting research and the T1D community. "The combined strengths of research, advocacy and community engagement are helping us support more members of the T1D community and enabling more people to support our mission," Dr. Kowalski said. "JDRF works to build partnerships that advance research and build and sustain critical support for type 1 diabetes (T1D) research funded by the Federal Government."

Expanding Access to Coverage:

Learn more about JDRF's research and advocacy priorities at jdrf.org and read the health insurance guide JDRF has complied to help the T1D community navigate their healthcare and health insurance here.

About JDRF

JDRF is the leading global organization funding type 1 diabetes (T1D) research. Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.2 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact and uniting on a national stage to pool resources, passion and energy. We collaborate with academic institutions, policymakers and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.

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JDRF Reaches New Milestones in Work to Drive Cures for Type 1 Diabetes and Improve Lives - PRNewswire

Dental caries occur when the acid from plaque erodes the tooth enamel and forms cavities. Caries is a common dental disease, prevalent in both developed and developing countries, affecting every stratum and age group of the society. The FISABIO Foundation discovered a new bacterial species named Streptococcus dentisani, which acts as a shield against caries-causing bacteria, producing a substance that inhibits them and prevents caries. Streptococcus dentisani bacteria is used as a preventive probiotic product to improve oral health in the food and cosmetic industries (mouthwash, toothpaste). Probiotic solutions are expected to be a groundbreaking approach toward anticaries treatment as these solutions have the potential to improve health and reduce health care expenditure. Anticaries treatment is considered an attractive concept for next-generation anticavity therapy, as it has the potential to prevent tooth replacement or other dental surgical replacements required due to cavity formation. The use and availability of different types of anticaries treatments have evolved over the past century; however, further research is ongoing in order to develop its more clinical applications and reduce the adverse effects associated with the use of anticaries treatments.

A major factor driving the anticaries treatments market is the high incidence of dental problems globally. Rise in incidence of periodontics among young adults and increase in demand for stem cell anticaries treatment techniques, especially among the geriatric population, are the other factors anticipated to drive the anticaries treatments market during the forecast period. Favorable price of anticaries products and emergence of new technologies using an anticaries product as a functional food, mouthwash, toothpaste, chewing gum, etc. are expected to propel the global anticaries treatments market. According to the World Health Organization, complete loss of teeth affects approximately 30% of the geriatric population in the age group of 65-74 years. However, prevalence rates of anticaries are increasing in low and middle income countries. Factors, such as, preference for endodontic treatment over anticaries treatment in major dental surgeries and local inflammatory activity which results in chronic complications and dental replacements are projected to restrain the growth of the anticaries treatments market during the forecast period.

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The global anticaries treatments market can be segmented on the basis of product type, end-user, and region. In terms of product type, the anticaries treatments market can be categorized into topical solution, gel-based, foam-based, toothpaste, and others. The gel-based segment accounted for a prominent share of the global anticaries treatments market in 2017, due to increasing prevalence of dental surgery and rising demand for cosmetic surgery, especially in the emerging economies, such as, China, Brazil, and India. In terms of end-user, the anticaries treatments market can be divided into cosmetic industry, home care, dental clinics, and others.

In terms of region, the global anticaries treatments market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is projected to dominate the global anticaries treatments market during the forecast period due to increasing dental services and stem cell research in the region. According to the Dental Health Services, in 2015, total expenditure on dental services in Canada amounted to US$ 13.6 Bn. Expenditure of the private sector was estimated at US$ 12.7 Bn, while that of the public sector at US$ 846 Mn. In April 2017, Unilever launched an in-clinic remineralisation regime to regenerate professionally advanced enamel serum. The Unilever brand claimed 82% of the enamel mineral regenerated after 3 days. Furthermore, increasing prevalence of dental cavities and periodontics, especially in the developing countries, such as, China and India has boosted the demand for dental surgery. According to the World Health Organization, nearly 60% to 90% of school children and nearly 100% of adults have dental cavities. The incidence of dental surgery, general prosthetic fixation, periodontal inflammation, and other dental diseases is increasing in Asia Pacific. This, in turn, is anticipated to fuel the demand for cost-effective aesthetic and dental surgeries. These factors are projected to drive the anticaries treatments market in Asia Pacific between 2018 and 2026.

Key players operating in the global anticaries treatments market include Unilever, Ocata Therapeutics, ICPA Health, GlaxoSmithKline plc, and Church & Dwight, Inc.

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MRR.BIZ has been compiled in-depth market research data in the report after exhaustive primary and secondary research. Our team of able, experienced in-house analysts has collated the information through personal interviews and study of industry databases, journals, and reputable paid sources.

The report provides the following information: Tailwinds and headwinds molding the market trajectory Market segments based on products, technology, and applications Prospects of each segment Overall current and possible future size of the market Growth pace of the market Competitive landscape and key players strategies

The main aim of the report is to: Enable key stakeholder in the market bet right on it Understand the opportunities and pitfalls awaiting them Assess the overall growth scope in the near term Strategize effectively with respect to production and distribution

MRR.BIZ is a leading provider of strategic market research. Our vast repository consists research reports, data books, company profiles, and regional market data sheets. We regularly update the data and analysis of a wide-ranging products and services around the world. As readers, you will have access to the latest information on almost 300 industries and their sub-segments. Both large Fortune 500 companies and SMEs have found those useful. This is because we customize our offerings keeping in mind the specific requirements of our clients.

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Anticaries Treatments Market to Witness a Healthy Y-o-Y Growth during 2018 2026 - Jewish Life News

Over the last two decades, few drugs have been approved for the treatment of AML, however increased understanding of the leukemic genome has allowed the development of targeted therapeutic agents.2,3 Between April 2017 and November 2018, eight new drugs in various different classes were approved, including: enasidenib, ivosidenib, midostaurin, glasdegib, venetoclax, and gilteritinib.4 An overview of mutations that can be targeted in AML is available on the AML Global Portal (AGP).

Some of these targeted agents are being investigated in novel combination therapies, as different lines of AML treatment, and some as monotherapy compared to standard chemotherapy. This article provides a summary of AGP content relating to novel agent-containing combinations to date and introduces the new editorial theme of Novel combination therapies using targeted agents.

The AGP were pleased to speak to Andrew Wei during the European School of Haematology (ESH) Translational Research Conference on AML, 2019, about how targeted therapies will be used in the future treatment of AML. In this interview, available below, Andrew Wei discusses whether these targeted agents should be used in combination or sequentially.

During the American Society of Clinical Oncology (ASCO) meeting in 2018, the AGP spoke to Uma Borate about how targeted therapies will impact the treatment of AML. Uma Borate discussed targeted agents, such as venetoclax, azacitidine, ivosidenib, and enasidenib.

Precision medicine may have a particular role in the treatment of older patients, since this population are often unable to receive allo-HSCT or intensive chemotherapy regimens. During the 61st American Society of Hematology (ASH) meeting, the AGP spoke to John Byrd about how precision medicine may be used to improve the outcomes of older patients with AML.

The AGP interviewed Courtney DiNardo about the treatment of elderly or frail patients with AML at the 1st National Cancer Research Institute (NCRI) AML Academy Meeting. Watch Courtney DiNardo discuss how the treatment approach for this patient group has changed in recent years with the advent of combination therapies below.

Studies have shown that epigenetic dysregulation can promote a preleukemic state that precedes leukemic transformation. Drugs targeting epigenetic dysregulation have been developed and include: azacitidine and decitabine, which are hypothesized to reverse DNA hypermethylation and reactivate repressed tumor suppressor genes; ivosidenib and enasidenib, which target IDH1 and IDH2 mutations thought to be involved in aberrant DNA hypermethylation.2 Combining these agents may improve patient outcome compared to azacitidine monotherapy.

Ivosidenib/enasidenib plus azacitidine: phase I/II study: AG221-AML-005 (NCT02677922)5,6,7

Patients with newly diagnosed AML, with either IDH1 mutations (mIDH1) or IDH2 mutations (mIDH2), were treated with azacitidine plus ivosidenib (for mIDH1) or enasidenib (for mIDH2).

Ivosidenib/enasidenib plus standard chemotherapy8

A phase III study, HOVON 150/AMLSG 29-18, is comparing enasidenib or ivosidenib (by mIDH status) plus chemotherapy (7+3) versus placebo.9 An overview of IDH inhibitors, including further information on the data from trials involving these combination therapies, is available here.

Other new targets for AML therapies are the FLT3 pathways. FLT3 is a transmembrane tyrosine kinase with two mutational subtypes: an internal tandem duplication (ITD) or point mutations in the tyrosine kinase domain (TKD).4 A third of patients with de novo AML have mutations in the FLT3 gene and patients with FLT3-ITD AML have a poor prognosis. FLT3 is therefore an attractive target for therapeutic agents, with several FLT3 inhibitors in clinical development, such as sorafenib, midostaurin, quizartinib, crenolanib, and gilteritinib.2,4

Examples of combination therapies involving FLT3 inhibitors are shown in Table 1 below. One example of a trial involving a FLT3 inhibitor is the RATIFY (NCT00651261) trial. The RATIFY trial assessed whether the addition of midostaurin to standard chemotherapy could prolong survival in newly diagnosed adult patients with FLT3-ITD AML compared to placebo + chemotherapy. The results showed midostaurin prolonged median OS and event-free survival (EFS) compared to placebo10:

However, the authors questioned if more specific FLT3 inhibitors could further improve outcomes.10

Table 1. Examples of combination therapies containing FLT3 inhibitors4,11,12

Targeted agent

In combination with/versus

Patient population

Trial name/NCT reference

Midostaurin

Induction chemo

ND AML with FLT3-ITD or TKD mutations

RATIFY

NCT00651261

Quizartinib

Induction chemo

ND AML with FLT3-ITD mutations

QuANTUM-First

NCT02668653

Crenolanib

vs midostaurin, in combination with induction chemo

ND AML with FLT3-ITD mutations

NCT03258931

Gilteritinib

Induction chemo

ND AML

NCT02310321

Sorafenib

Induction chemo

ND AML <60 years old

SORAML

NCT00893373

Learn more about FLT3 inhibitors in AML here.

During the 2019 EHA meeting, the AGP spoke to Alexander Perl about gilteritinib for the treatment of relapsed/refractory (R/R) AML.

Another example of a targeted agent for the treatment of AML is venetoclax, which is a Bcl-2 inhibitor. Venetoclax is approved by the U.S. Food & Drug Administration (FDA) for the treatment of newly diagnosed patients with AML who are not eligible for intensive induction chemotherapy, in combination with azacitidine, decitabine, or low dose cytarabine.13

Results from a phase Ib study (NCT02203773) investigating venetoclax with decitabine or azacitidine showed that these combination therapies led to a 61% complete remission or complete remission with incomplete marrow recovery rate in newly diagnosed patients with AML aged 65 and over.14 A phase III study (NCT02993523) comparing venetoclax plus azacitidine to azacitidine alone in patients with treatment-nave AML is ongoing.15

During the Acute Leukemias XVII Biology and Treatment Strategies biennial symposium, Andrew Wei gave a presentation on novel therapeutic combinations containing venetoclax for the treatment of AML.

There are a number of ongoing studies of venetoclax in combination therapies and examples of these include4:

During the 2018 ASCO meeting, the AGP spoke to Courtney DiNardo about the combination of venetoclax with hypomethylating agents (HMAs).

Subsequently, during the Society of Hematologic Oncology (SOHO) meeting in 2019, the AGP spoke to Jeffrey Lancet about the potential of venetoclax + HMAs to replace induction chemotherapy.

The AGP spoke to Marina Konopleva at the 2019 EHA meeting about Bcl-2 as a universal target in AML.

The development of targeted therapies and novel combinations holds promise for the treatment of AML, especially when compared to currently approved options. In some cases, these combinations may be more efficacious than intensive induction chemotherapy. In addition, some patients have been able to undergo allo-HSCT following treatment with a novel combination, which may indicate that the future of AML treatment will be in novel combinations and involve less chemotherapy. Additionally, these combinations appear tolerable to date.

Despite the promising results seen so far, not all patients respond equally. In order to maximize response to these therapies, it will be important to identify the right patient subgroups, with specific mutations, and may require particular combinations of agents to be used.2

Read this article:
An introduction to novel combination therapies using targeted agents in AML - AML Global Portal

Meticulous Research leading global market research company published a research report titled Medical Aesthetics Market by Product (Facial Aesthetics, Cosmetic Implants, Skin Aesthetic Devices, Thread Lift Products, Body Contouring Devices, Hair Removal Devices), End User (Hospitals, Medical Spas, Home Care Settings)Global Forecast to 2025.

According to this latest publication from Meticulous Research, the global medical aesthetics market is expected to grow at a CAGR of 11.5% from 2019 to reach $22.2 billion by 2025. The growth of this market will be driven by factors such as increasing adoption of minimally invasive and noninvasive aesthetic procedures, increasing public awareness about cosmetic procedures, and rising adoption of medical aesthetic procedures among geriatric population to improve their appearance. However, risk and complications associated with medical aesthetic procedures may hamper the growth of the market to a certain extent.

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Key questions answered in the report-Which are the high growth market segments in terms of products, technology, disease type, end user, and region/countries?What was the historical market for medical aesthetics market across the globe?What are the market forecasts and estimates for the period 2018-2025?What are the major drivers, opportunities, and challenges in the global medical aesthetics market?Who are the major players in the global medical aesthetics market?How is the competitive landscape and who are the market leaders in the global medical aesthetics market?What are the recent developments in the global medical aestheticsmarket?What are the different strategies adopted by the major players in the global medical aesthetics market?What are the geographical trends and high growth regions/ countries?Who are the local emerging players in the global medical aesthetics market and how do they compete with the global players?

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The global medical aesthetics market study presents historical market data in terms of values (2017 and 2018), estimated current data (2019), and forecasts for 2025- by product (facial aesthetics, cosmetic implants, skin aesthetic devices, physician-dispensed cosmeceuticals and skin lighteners, thread lift products, body contouring devices, hair removal devices, tattoo removal devices, and nail treatment laser devices), and end user (hospitals, clinics, and medical spas, beauty centers, and home care settings). The study also evaluates industry competitors and analyzes the market at a regional and country level.

On the basis of product type, facial aesthetics market segment is estimated to command the largest share of the global medical aesthetics market in 2019; whereas, cosmetic implants segment is expected to grow at the highest CAGR during the forecast period due to increasing growth of minimally invasive reconstruction surgeries, technological advancements such as injectable fillers and gummy bear breast implants, rising number of congenital face disorders, and increasing awareness about aesthetic appearance. A comprehensive range of cosmetic plastic surgery techniques and implants have been developed over the years for improving the appearance or restoring function to the human body. The implants used in cosmetic procedures are to enhance the aesthetic looks of an individual and rectify the deformities caused due to accidents, trauma, and congenital disorders.

On the basis of end user, hospitals, clinics, and medical spas segment is estimated to hold the largest share of the global medical aesthetics market in 2019. Hospitals, clinics, and medical spas are typically well-equipped with technologically advanced instruments/devices and have skilled professionals to provide effective cosmetic treatment to its patients. This has led to their greater share in the global medical aesthetics market. Hospital and medical spas design their services to improve the aesthetic procedures for patient well-being. Medical aesthetic services involve highly advanced technology that needs the combination of healthcare and beauty services. In aesthetic treatment, advanced technologies are increasingly being used to provide medical procedures designed to offer significant cosmetic change for patients. The growth of the hospitals, clinics, and medical spas segment in the market is primarily driven by the growing number of patients undergoing cosmetic procedures to enhance self-esteem, increasing healthcare expenditure on cosmetic procedures, growing geriatric population, and greater uptake of technologically advanced medical aesthetics devices in the hospitals and clinics to provide effective treatments to the patients.

Browse key industry insights spread across 195 pages with 185 market data tables & 18 figures & charts from the market research report:https://www.meticulousresearch.com/product/medical-aesthetics-market-5028/

Geograhic Review:

This research report analyzes major geographies and provides comprehensive analysis of North America (U.S., Canada), Europe (Germany, France, Italy, Spain, and U.K.), Asia-Pacific (China, India, South Korea, Japan, and Australia), Latin America, and Middle East & Africa. North America commanded the largest share of the global medical aesthetics market, followed by Europe and Asia Pacific. The largest share of North American region in the medical aesthetics market is primarily attributed to the growing healthcare sector, increasing awareness and adoption of aesthetic procedures among the population, growing healthcare expenditure, rising incidences of skin diseases, growing geriatric population, various technological advancements, and increase in the consciousness about physical appearances.

Asia Pacific region is expected to be the fastest growing geographic markets for medical aesthetics devices with countries, such as China, Japan, South Korea and India among others for being the largest contributors to the growth of the market. Additionally, rapid urbanization, increasing investments by healthcare providers towards infrastructure improvement, increasing awareness among the population, growing beauty consciousness, and availability of increasing range of advanced products and technologies is contributing to the growth of the medical aesthetics market in the Asia Pacific region.

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Key players:

The major players operating in the globalmedical aesthetics marketare Allergan plc (Ireland), Alma Lasers (Israel), Anika Therapeutics, Inc. (US), Cutera, Inc. (US), Cynosure Inc. (US), El.En. S.P.A. (Italy), Fotona D.O.O (Solvenia& US), Galderma Laboratories, L.P. (US), Mentor Worldwide LLC (US), and Merz Aesthetics (Germany) among others.

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Surgical Sutures Market by Product (Suture Thread (Synthetic, Nylon, Silk, Prolene, Steel), Automatic Suture Device), Application (CVD, General, Orthopedic, Gynec, Ophthalmic, Plastic, Cosmetics), End User (Hospitals, ASC, Clinic) Global Forecast to 2024

Flow Cytometry Market by Product (Cell Analyzers, Cell Sorter, Software, Reagents), Technology (Cell Based, Bead Based), Application (Drug Discovery, Stem Cell Research, Cancer, Organ Transplant, Commercial) and by End-user Global Forecast to 2027

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Medical Aesthetics Market Is Expected To Grow At CAGR Of 11.5% From 2019 To Reach $22.2 Billion By 2 - PharmiWeb.com

DetailsCategory: Small MoleculesPublished on Wednesday, 12 February 2020 17:52Hits: 446

PLEASANTON, CA, USA I February 11, 2020 I Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Japan, today announced that the U.S. FDA has accepted for Priority Review its NDA for oral C-DEC (cedazuridine and decitabine) as a treatment for adults with previously untreated intermediate- and high-risk MDS including CMML. The NDA submission is based on data from the ASCERTAIN phase 3 study which evaluated the 5-day decitabine exposure equivalence of oral C-DEC and IV decitabine.

We are very pleased that the FDA has accepted our NDA for Priority Review, said Dr Mohammad Azab, MD, president & chief medical officer of Astex Pharmaceuticals, Inc. Subject to FDA review and regulatory approval, oral C-DEC may offer a new option for patients with MDS and CMML that saves them the burden of 5-day IV infusions every month during their treatment period. We are grateful to all the patients, investigators and other healthcare providers, and partner research and manufacturing organizations, who contributed to the clinical development program of oral C-DEC.

The FDA grants Priority Review to applications for drugs that, if approved, would provide significant improvements in the safety and effectiveness of the treatment, diagnosis or prevention of serious conditions. The Priority Review designation means FDAs goal is to take action on an NDA application within six months (compared to the ten months under standard review).

Oral C-DEC is an investigational compound and is not currently approved in any country.

Astexs parent company, Otsuka Pharmaceutical Co., Ltd., and Taiho Pharmaceutical Co., Ltd. previously announced that, subject to regulatory approvals, commercialization of oral C-DEC in the U.S. and Canada will be conducted by Taiho Oncology, Inc. and Taiho Pharma Canada, Inc. respectively. Astex, Otsuka and Taiho are all members of the Otsuka group of companies.

About C-DEC (Cedazuridine 100 mg and Decitabine 35 mg) Fixed-Dose Combination

C-DEC is a novel, orally administered fixed dose combination of cedazuridine, an inhibitor of cytidine deaminase,1 with the anti-cancer DNA hypomethylating agent, decitabine.2 By inhibiting cytidine deaminase in the gut and the liver, C-DEC is designed to allow for oral delivery of the approved DNA hypomethylating agent, decitabine, at exposures which emulate exposures achieved with the approved intravenous form of decitabine administered over 5 days.3

C-DEC has been evaluated in a phase 1/2 pharmacokinetics-guided dose escalation and dose confirmation study in patients with MDS and CMML (see https://www.clinicaltrials.gov NCT02103478) and a pivotal phase 3 study (ASCERTAIN) (see https://www.clinicaltrials.gov NCT03306264) conducted at investigator sites in the US and Canada and designed to confirm the results from the phase 1/2 study. The phase 3 study is now being extended to include patients with acute myeloid leukemia (AML) unsuitable to receive intensive induction chemotherapy.

In September 2019 Astex announced that C-DEC had received orphan drug designation for the treatment of MDS and CMML from the U.S. FDA.

The concept of using cedazuridine to block the action of cytidine deaminase is also being evaluated in a low dose formulation of cedazuridine and decitabine for the treatment of lower risk MDS (see https://www.clinicaltrials.gov NCT03502668).

About the Phase 3 ASCERTAIN Study

The study was designed as a randomized crossover study comparing oral C-DEC (cedazuridine 100 mg and decitabine 35 mg fixed-dose combination tablet given once daily for 5 days on a 28-day cycle) to IV decitabine (20 mg/m2 administered as a daily, 1-hour IV infusion for 5 days on a 28-day cycle) in the first 2 cycles with patients continuing to receive oral C-DEC from Cycle 3 onwards. The data from the ASCERTAIN study was presented at the American Society of Hematology (ASH) Meeting in Orlando, Florida in December 2019 by Dr Guillermo Garcia-Manero, MD, professor and chief of section of myelodysplastic syndromes, Department of Leukemia at The University of Texas MD Anderson Cancer Center, on behalf of the study investigators.4 The data demonstrated that the ASCERTAIN study met the primary endpoint of total 5-Day decitabine Area-Under-The-Curve (AUC) equivalence of oral C-DEC and IV decitabine. Safety findings from the study were consistent with those anticipated with IV decitabine, with no significant differences in the incidence of most common adverse events between oral C-DEC and IV decitabine in the first 2 randomized cycles. The most common adverse events of any grade >20% regardless of causality in patients in the first 2 randomized cycles who received oral C-DEC were thrombocytopenia (43.8%), neutropenia (35.4%), anemia (36.9%), and fatigue (23.8%). The ASH presentation can be downloaded from the Astex website at https://astx.com/media-center/presentations-and-publications/ASTX727 ASCERTAIN Presentation - ASH - December 2019

About Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)

Myelodysplastic syndromes are a heterogeneous group of hematopoietic stem cell disorders characterized by dysplastic changes in myeloid, erythroid, and megakaryocytic progenitor cells, and associated with cytopenias affecting one or more of the three lineages. U.S. incidence of MDS is estimated to be 10,000 cases per year, although the condition is thought to be under-diagnosed.5,6 The prevalence has been estimated to be from 60,000 to 170,000 in the U.S.7 MDS may evolve into acute myeloid leukemia (AML) in one-third of patients.8 The prognosis for MDS patients is poor; patients die from complications associated with cytopenias (infections and bleeding) or from transformation to AML. CMML is a clonal hematopoietic malignancy characterized by accumulation of abnormal monocytes in the bone marrow and in blood. The incidence of CMML in the U.S. is approximately 1,100 new cases per year,9 and CMML may transform into AML in 15% to 30% of patients.10 The hypomethylating agents decitabine and azacitidine are effective treatment modalities for hematologic cancers and are FDA-approved for the treatment of higher-risk MDS and CMML. These agents are administered by IV infusion, or by large-volume subcutaneous injections.

About Astex Pharmaceuticals, Inc.

Astex is a leader in innovative drug discovery and development, committed to the fight against cancer. Astex is developing a proprietary pipeline of novel therapies and has multiple partnered products in development under collaborations with leading pharmaceutical companies. Astex is a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Tokyo, Japan.

Otsuka is a global healthcare company with the corporate philosophy: Otsukapeople creating new products for better health worldwide. Otsuka researches, develops, manufactures and markets innovative and original products, with a focus on pharmaceutical products for the treatment of diseases and nutraceutical products for the maintenance of everyday health.

For more information about Astex Pharmaceuticals, Inc. please visit: http://www.astx.com

For more information about Otsuka Pharmaceutical, please visit: http://www.otsuka.com/en/

For more information about Taiho Pharmaceutical, please visit: https://www.taihooncology.com/

References

SOURCE: Astex Pharmaceuticals

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Astex Pharmaceuticals Announces US Food and Drug Administration (FDA) Acceptance for Review of an NDA for the Combination Oral Hypomethylating Agent...

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